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Phase II Randomized Trial of Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

This study is currently recruiting participants.
Verified by FDA Office of Orphan Products Development, May 1999

Sponsors and Collaborators: FDA Office of Orphan Products Development
Boston University
Information provided by: FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier: NCT00004412
  Purpose

OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers.

II. Determine the effect of arginine butyrate therapy on tissue factors related to promotion or inhibition of wound healing in these patients.

III. Determine whether the regimen used in this study is appropriate for testing in pivotal trials.


Condition Intervention Phase
Skin Ulcers
Sickle Cell Anemia
Drug: arginine butyrate
Phase II

Genetics Home Reference related topics:   beta thalassemia    sickle cell disease   

MedlinePlus related topics:   Anemia    Sickle Cell Anemia   

Drug Information available for:   Arginine    Arginine hydrochloride    Arginine butyrate   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:   30
Study Start Date:   September 1997

Detailed Description:

PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to one of two treatment arms. Arm I: Patients receive arginine butyrate IV over 6-9 hours at night 5 days a week for 12 weeks, plus concurrent standard local therapy consisting of cleaning, saline irrigation, and dressing changes as prescribed by each patient's physician. Patients who experience progressive healing receive arginine butyrate 3-4 times a week. Arginine butyrate treatment may be discontinued and reinstated following a single 2 week medical complication.

Arm II: Patients receive standard local therapy only for 12 weeks. Patients randomized to arm II may cross over to receive arginine butyrate if no or less than 25% healing is observed after 12 weeks.

Patients whose ulcers have closed by at least 15% per course may receive 2 additional 8-week courses of arginine butyrate therapy and are followed for 2 months after healing is completed.

  Eligibility
Ages Eligible for Study:   16 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Significant sickle cell syndrome including HbSS, S-beta thalassemia, and hemoglobin variants Lower extremity or ankle ulcer (or ulcers) present for at least 6 months without healing --Prior/Concurrent Therapy-- Biologic therapy: No chronic transfusion therapy Chemotherapy: No prior or concurrent cancer chemotherapy No concurrent butyrate derivatives Prior treatment with hydroxyurea allowed if on a stable dose for at least 1 year Endocrine therapy: No concurrent corticosteroid therapy Radiotherapy: Not specified Surgery: Not specified Other: Must be treated with antibiotics prior to entry for complicating cellulitis or secondary infections --Patient Characteristics-- Age: 16-60 Performance status: Not specified Hematopoietic: Not specified Hepatic: No hepatic compromise Transaminases no greater than 250 IU Renal: No renal compromise Creatinine no greater than 1.2 mg/mL (adults) Creatinine no greater than 0.9 mg/mL (teenagers) Other: Not pregnant Fertile patients must use effective contraception No poorly controlled seizure disorders No other secondary conditions that might inhibit immune function

  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004412

Locations
United States, Illinois
University of Illinois College of Medicine     Recruiting
      Chicago, Illinois, United States, 60612
      Contact: Mabel Koshy     312-996-5680        
United States, Massachusetts
Boston University School of Medicine     Recruiting
      Boston, Massachusetts, United States, 02118
      Contact: Susan Park Perrine     617-638-4173        
United States, New York
Mount Sinai School of Medicine     Recruiting
      New York, New York, United States, 10029
      Contact: George F. Atweh     212-785-6662        
United States, Tennessee
University of Tennessee, Memphis Cancer Center     Recruiting
      Memphis, Tennessee, United States, 38103
      Contact: Patricia E. Adams-Graves     901-448-5798        

Sponsors and Collaborators

Investigators
Study Chair:     Douglas V. Faller     Boston University    
  More Information


Study ID Numbers:   199/13302, BUSM-FDR001376, BUSM-3889
First Received:   October 18, 1999
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00004412
Health Authority:   United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
dermatologic disorders  
genetic diseases and dysmorphic syndromes  
hematologic disorders  
rare disease  
sickle cell anemia
skin ulcers
thalassemia major

Study placed in the following topic categories:
Skin Diseases
Hematologic Diseases
Ulcer
Beta-thalassemia
Anemia
Rare Diseases
Anemia, Hemolytic
Thalassemia
Sickle cell anemia
Anemia, Hemolytic, Congenital
Thalassemia minor
Genetic Diseases, Inborn
Beta-Thalassemia
Hemoglobinopathies
Arginine butyrate
Hemoglobinopathy
Skin Ulcer
Anemia, Sickle Cell

Additional relevant MeSH terms:
Pathologic Processes
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on November 20, 2008




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